Parents in tears of joy over £1.8m drug

HEALTH: The Yorkshire parents of a 10-month-old baby say they were overwhelmed and cried when a treatment which could keep him alive was approved for use by the NHS.

Rosie-Mae Walton, 19, and Wes Powell, 22, hope their son Marley, who has a rare genetic disorder, will be eligible for the drug costing £1.8m a dose.

THE Yorkshire parents of a 10-month-old baby say they were overwhelmed and cried when they heard that the treatment which could keep him alive had been approved for use by the NHS despite it being labelled the most expensive drug in the world.

Rosie-Mae Walton, 19, and Wes Powell, 22, said “it would mean the absolute world to us” if their son Marley, who has a rare genetic disorder, was eligible for treatment with the US gene therapy Zolgensma after it was licensed for use on the NHS in England.

But the couple from Driffield, East Yorkshire, said it is too early to know whether he will be approved for the drug.

Ms Walton and Mr Powell have been raising money for Marley, who has Type 1 spinal muscular atrophy (SMA), to go to the United States for treatment with Zolgensma which has an eye-watering price tag of around £1.8m for the single dose that is needed.

Now the family’s hopes have been raised by the landmark news from the NHS last week.

Ms Walton told the PA news agency: “Our initial reaction when Zolgensma was approved by NHS England was shock and we did cry with being so overwhelmed.

“It’s so amazing that it’s been approved and can help so many other babies that are diagnosed with SMA.

“We are still unsure if Marley is eligible for Zolgensma and are still waiting on news from our specialists before any decisions can be made.

“We hope that he will be. However, until then we do not know.”

Ms Walton added: “I’ve been in touch with some of Marley’s specialists and they have just confirmed about it being approved. However, it’s just a waiting game for them as much as it is us at this moment in time.

“It would mean the absolute world to us if Marley was to get Zolgensma treatment.

“He deserves the world and we hope we can give him the treatment to give him the life he deserves and live his life healthier.”

NHS England said last week that it had struck a “landmark confidential deal” with US-based manufacturer Novartis Gene Therapies ensuring patients can get the crucial treatment “at a price that is fair to taxpayers”.

NHS chief executive Sir Simon Stevens said the deal was a “lifechanger” for children and their families.

Around 95 per cent of babies with SMA do not live past 18 months without effective treatment. The condition is similar to motor neurone disease, and makes Marley’s muscles extremely weak, stops him moving his arms and legs, and causes breathing and numerous other life-limiting difficulties.

Studies have shown a one-time intravenous infusion of Zolgensma can help youngsters breathe without ventilators, sit up without help, and crawl and walk.

As many as 80 babies and young children could benefit from the life-changing treatment each year, NHS England has said.

The drug contains a replica of the missing gene SMN1 and the active ingredient enters the nerves and restores the gene.

Our initial reaction was shock and we did cry.

Rosie-Mae Walton, mother of Marley who has Type 1 spinal muscular atrophy.